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WO2013032643A3 - Lipids capable of conformational change and their use in formulations to deliver therapeutic agents to cells - Google Patents

Lipids capable of conformational change and their use in formulations to deliver therapeutic agents to cells Download PDF

Info

Publication number
WO2013032643A3
WO2013032643A3 PCT/US2012/049822 US2012049822W WO2013032643A3 WO 2013032643 A3 WO2013032643 A3 WO 2013032643A3 US 2012049822 W US2012049822 W US 2012049822W WO 2013032643 A3 WO2013032643 A3 WO 2013032643A3
Authority
WO
WIPO (PCT)
Prior art keywords
cells
formulations
therapeutic agents
conformational change
deliver therapeutic
Prior art date
Legal status (The legal status is an assumption and is not a legal conclusion. Google has not performed a legal analysis and makes no representation as to the accuracy of the status listed.)
Ceased
Application number
PCT/US2012/049822
Other languages
French (fr)
Other versions
WO2013032643A2 (en
Inventor
Sujit Kumar Basu
Current Assignee (The listed assignees may be inaccurate. Google has not performed a legal analysis and makes no representation or warranty as to the accuracy of the list.)
Dicerna Pharmaceuticals Inc
Original Assignee
Dicerna Pharmaceuticals Inc
Priority date (The priority date is an assumption and is not a legal conclusion. Google has not performed a legal analysis and makes no representation as to the accuracy of the date listed.)
Filing date
Publication date
Application filed by Dicerna Pharmaceuticals Inc filed Critical Dicerna Pharmaceuticals Inc
Publication of WO2013032643A2 publication Critical patent/WO2013032643A2/en
Publication of WO2013032643A3 publication Critical patent/WO2013032643A3/en
Anticipated expiration legal-status Critical
Ceased legal-status Critical Current

Links

Classifications

    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N15/00—Mutation or genetic engineering; DNA or RNA concerning genetic engineering, vectors, e.g. plasmids, or their isolation, preparation or purification; Use of hosts therefor
    • C12N15/09—Recombinant DNA-technology
    • C12N15/87—Introduction of foreign genetic material using processes not otherwise provided for, e.g. co-transformation
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K47/00—Medicinal preparations characterised by the non-active ingredients used, e.g. carriers or inert additives; Targeting or modifying agents chemically bound to the active ingredient
    • A61K47/50—Medicinal preparations characterised by the non-active ingredients used, e.g. carriers or inert additives; Targeting or modifying agents chemically bound to the active ingredient the non-active ingredient being chemically bound to the active ingredient, e.g. polymer-drug conjugates
    • A61K47/51—Medicinal preparations characterised by the non-active ingredients used, e.g. carriers or inert additives; Targeting or modifying agents chemically bound to the active ingredient the non-active ingredient being chemically bound to the active ingredient, e.g. polymer-drug conjugates the non-active ingredient being a modifying agent
    • A61K47/62—Medicinal preparations characterised by the non-active ingredients used, e.g. carriers or inert additives; Targeting or modifying agents chemically bound to the active ingredient the non-active ingredient being chemically bound to the active ingredient, e.g. polymer-drug conjugates the non-active ingredient being a modifying agent the modifying agent being a protein, peptide or polyamino acid
    • A61K47/64—Drug-peptide, drug-protein or drug-polyamino acid conjugates, i.e. the modifying agent being a peptide, protein or polyamino acid which is covalently bonded or complexed to a therapeutically active agent
    • A61K47/645—Polycationic or polyanionic oligopeptides, polypeptides or polyamino acids, e.g. polylysine, polyarginine, polyglutamic acid or peptide TAT
    • A61K47/6455—Polycationic oligopeptides, polypeptides or polyamino acids, e.g. for complexing nucleic acids
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K9/00—Medicinal preparations characterised by special physical form
    • A61K9/10—Dispersions; Emulsions
    • A61K9/127—Synthetic bilayered vehicles, e.g. liposomes or liposomes with cholesterol as the only non-phosphatidyl surfactant
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K48/00—Medicinal preparations containing genetic material which is inserted into cells of the living body to treat genetic diseases; Gene therapy

Landscapes

  • Health & Medical Sciences (AREA)
  • Life Sciences & Earth Sciences (AREA)
  • Chemical & Material Sciences (AREA)
  • Engineering & Computer Science (AREA)
  • Genetics & Genomics (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • General Health & Medical Sciences (AREA)
  • Veterinary Medicine (AREA)
  • Wood Science & Technology (AREA)
  • Pharmacology & Pharmacy (AREA)
  • Epidemiology (AREA)
  • Animal Behavior & Ethology (AREA)
  • Proteomics, Peptides & Aminoacids (AREA)
  • Public Health (AREA)
  • Molecular Biology (AREA)
  • Biochemistry (AREA)
  • Organic Chemistry (AREA)
  • Biomedical Technology (AREA)
  • Zoology (AREA)
  • Biotechnology (AREA)
  • General Engineering & Computer Science (AREA)
  • Medicinal Chemistry (AREA)
  • Microbiology (AREA)
  • Plant Pathology (AREA)
  • Biophysics (AREA)
  • Physics & Mathematics (AREA)
  • Dispersion Chemistry (AREA)
  • Medicinal Preparation (AREA)
  • Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)

Abstract

The invention provides lipid-based composition for use in the administration of therapeutic payloads to target cells or tissues in vivo. The lipid compounds of the invention include a peptide-based moiety which is induced to change its conformation upon entering a cell or intracellular compartment. The induced conformation change in the peptide-based moiety consequently triggers the efficient release of the payload from the lipid-based composition.
PCT/US2012/049822 2011-08-31 2012-08-07 Lipids capable of conformational change and their use in formulations to deliver therapeutic agents to cells Ceased WO2013032643A2 (en)

Applications Claiming Priority (2)

Application Number Priority Date Filing Date Title
US201161529422P 2011-08-31 2011-08-31
US61/529,422 2011-08-31

Publications (2)

Publication Number Publication Date
WO2013032643A2 WO2013032643A2 (en) 2013-03-07
WO2013032643A3 true WO2013032643A3 (en) 2013-04-25

Family

ID=47757113

Family Applications (1)

Application Number Title Priority Date Filing Date
PCT/US2012/049822 Ceased WO2013032643A2 (en) 2011-08-31 2012-08-07 Lipids capable of conformational change and their use in formulations to deliver therapeutic agents to cells

Country Status (1)

Country Link
WO (1) WO2013032643A2 (en)

Cited By (1)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US12257289B2 (en) 2020-04-23 2025-03-25 KaloCyte, Inc. Self-assembling oxygen carrier compositions

Families Citing this family (6)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
WO2015005431A1 (en) * 2013-07-12 2015-01-15 石原産業株式会社 Composition for transferring gene to cell
AU2017267634C1 (en) * 2016-05-16 2022-05-26 The Board Of Regents Of The University Of Texas System Cationic sulfonamide amino lipids and amphiphilic zwitterionic amino lipids
EP3548005A4 (en) 2016-11-29 2020-06-17 Puretech Health LLC EXOSOME FOR THE OUTPUT OF THERAPEUTIC ACTIVE SUBSTANCES
CN110200920B (en) * 2019-06-18 2022-03-08 东南大学 A kind of reduction-sensitive pharmaceutical composition and its preparation and application
CN113816990B (en) * 2021-03-22 2023-08-22 联宁（苏州）生物制药有限公司 Modified amino acids and their use in ADCs
WO2025217110A1 (en) * 2024-04-09 2025-10-16 The Board Of Trustees Of The University Of Illinois Lipid nanoparticles for drug delivery and methods for making and using the same

Citations (4)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
WO1997041834A1 (en) * 1996-05-08 1997-11-13 Nika Health Products Limited Cationic virosomes as transfer system for genetic material
US20060002991A1 (en) * 2002-02-19 2006-01-05 Frank Essler Ph-sensitive cationic lipids, and liposomes and nanocapsules containing the same
EP1426044B1 (en) * 1999-04-13 2006-02-08 Sigma-Tau Industrie Farmaceutiche Riunite S.p.A. Use of esters of L-carnitine or alkanoyl L-carnitines as cationic lipids for the intracellular delivery of pharmacologically active compounds
WO2011072292A2 (en) * 2009-12-11 2011-06-16 Dicerna Pharmaceuticals, Inc. Phase changing formulations of rna and rna derivatives

Patent Citations (4)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
WO1997041834A1 (en) * 1996-05-08 1997-11-13 Nika Health Products Limited Cationic virosomes as transfer system for genetic material
EP1426044B1 (en) * 1999-04-13 2006-02-08 Sigma-Tau Industrie Farmaceutiche Riunite S.p.A. Use of esters of L-carnitine or alkanoyl L-carnitines as cationic lipids for the intracellular delivery of pharmacologically active compounds
US20060002991A1 (en) * 2002-02-19 2006-01-05 Frank Essler Ph-sensitive cationic lipids, and liposomes and nanocapsules containing the same
WO2011072292A2 (en) * 2009-12-11 2011-06-16 Dicerna Pharmaceuticals, Inc. Phase changing formulations of rna and rna derivatives

Cited By (1)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US12257289B2 (en) 2020-04-23 2025-03-25 KaloCyte, Inc. Self-assembling oxygen carrier compositions

Also Published As

Publication number Publication date
WO2013032643A2 (en) 2013-03-07

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